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April 13, 2013
April 11, 2013
FOR IMMEDIATE RELEASE
Gene Therapy Developed for ALS Treatment: New Biotech Company Neuralgene Enters Evaluation Phase
Patent-pending PRCN-829 delivers multiple genes, including Factor H, neural growth factors and regulators of TDP-43 to treat neurodegenerative disease ALS.
BOGOTA, Colombia, April 12, 2013 – Startup biotechnology company Neuralgene (http://neuralgene.com) has announced that it will begin animal studies in May to evaluate the efficacy of PRCN-829, its new gene therapy agent for the treatment of amyotrophic lateral sclerosis (ALS). PRCN-829 is the first gene therapy for sporadic ALS.
Neuralgene’s neurotropic AAV-based gene therapy platform for the treatment of neurodegenerative diseases is based on the stem cell work performed by Jason Williams, M.D., founder and CEO of Neuralgene. “This technology addresses several key aspects of the underlying pathology of ALS,” said Leonardo Gonzalez, M.D., clinical researcher for Neuralgene. “In his stem cell work, Dr. Williams had identified that production of Factor H by fat-derived mesenchymal stem cells may be a key mode of action.”
The gene therapy is based on Dr. Williams’ discovery that certain proteins produced by stem cells inhibit the attack of ALS. During the development of the gene therapy, he added new targets: neural growth factors and a protein implicated in ALS named TDP-43. “When Dr. Williams demonstrated the concept behind stem cells and how to address the treatment of ALS using gene therapy, we immediately knew that this was a revolutionary new concept,” said Dr. Gonzalez.
The PRCN-829 gene therapy is designed to not only target gene delivery to the brain and spinal cord, but also to genetically engineer stem cells. The AAV9 viral vector delivers multiple genes, which include Factor H (a regulator of complement activity), neural growth factors and regulators of TDP-43, to the neural cells. Initial animal studies have demonstrated the safety of the gene therapy platform.
“The problem with stem cell therapy for ALS is that the results are generally partial and temporary,” stated Dr. Williams. “This is because the stem cells produce the growth factors and other proteins for a short period, but then cease. Several stem cell studies have confirmed this. Now with gene therapy, we can increase those factors by a millionfold or greater so that recuperation lasts for many years or maybe is even lifelong.”
“ALS is a complex disease with many different underlying causes,” continued Dr. Williams. “Our gene therapy will target several of the main underlying mechanisms related to ALS with the hopes of getting a good response in a larger group of patients. However, our platform is versatile, allowing us to change and add different target genes. We expect that soon we will be able to perform a detailed genetic analysis of the patient, identifying their exact underlying cause of ALS. Then we will be able to tailor the therapy to each individual patient.”
The company has partnered with several labs for the development of its patent-pending neurotropic AAV-based gene therapy for the treatment of neurodegenerative diseases such as ALS. In fact, Neuralgene partnered with Dr. Williams’ imaging and image guided treatment facility, Precision StemCell in Bogota, to begin studies using image-guided administration of gene therapy to the spinal cord.
“This is a completely new therapy for ALS, and the groundwork for this technology will lead to the treatment of many other diseases,” said Dr. Williams. Neuralgene has several other AAV gene therapies in its research and development pipeline for the treatment of neurodegenerative diseases such as Parkinson’s and Multiple Sclerosis (MS). After initial testing of PRCN-829 in Colombia, Neuralgene plans to seek approval from the FDA for trials in the United States.
For more information, contact Dr. Leonardo Gonzalez at firstname.lastname@example.org.
Neuralgene is a startup biotech company focused on AAV gene therapy, which produces multiple proteins for the treatment of neurodegenerative disorders such as ALS and Parkinson’s. PRCN-829 is a patent-pending gene therapy designed to transmit genes of interest into neural and glial cells. The technology is based on discoveries made by Dr. Jason Williams while using autologous adipose derived stem cells in the treatment of ALS. Information constitutes “forward looking statements.”
Dr. Leonardo Gonzalez
Colombia: 57 (311) 231-3684
Dr. Gonzalez is a board certified regenerative medicine specialist from Bogota, Colombia. His research focus is in stem cell therapy and cancer. Dr. Gonzalez is the President for the Regenerative Medicine Society of Latin American. His current research interest is in neural reprogramming of adipose derived stem cells. He is also interested in the use of stem cells as a drug delivery system for the treatment of cancer. Dr. Gonzalez received his medical degree from National University, Bogota, Colombia.
March 28, 2013
Just Posted by Eric:
March 21, 2013
March 25, 2013
I would like to clarify with people about the move to Bogota. My clinic in Gulf Shores, Alabama, USA will still
be here. I will mainly be working here in Gulf Shores. I have over the past couple of years
been informed by some of my cancer colleagues that research in Bogota seemed very promising.
Several of them have found Colombia to be a good site to perform research as it is faster and less expensive.
I will be going there about 1 week a month to be involved in both cancer, gene therapy, stem cell and ALS
research. So, if there is concern that I am moving to Bogota to avoid something, that is not true.
I will mostly be in the USA performing my regular practice, minus stem cells. I am truly passionate
about gene therapy and stem cells, but I also have to keep my focus on my regular practice as well.
I feel that our research is moving in the right direction. When people ask about publishing data, don't worry,
that is the direction we are going. My practice was not set up to handle research. We have only been involved with
ALS for 1 year now. I have to fund any work myself, which takes time. I am currently trying to assemble a team
of researchers as well as our collaboration with the researchers in Colombia. Once we have the team together, expect
research papers to follow very quickly.
Further, people question, why Bogota, Colombia. First it is not what you think. Colombia is a great country with
very nice people. Second, they are very open to stem cells and gene therapy. I spoke at a meeting in Bogota filled with many doctors, they
were truly excited. They want to help, they want to be involved. The university is progressive and wants to be a world leader in
stem cell and gene therapy. I think that is reason enough. It is nice to be in a place where people see things the same way
that you do. That is why I like Colombia.
I did not expect to find myself in this world of ALS, but I have always been a fighter for a cause considered by many to be hopeless. I will
not give up, I expect to find a cure.
copy of Letter sent to Dr. Williams patients
Date: Mar 21, 2013 9:35 AM
Most of you have met me, as I work closely with Dr. Williams. I have had the opportunity to meet almost everyone during their time in Gulf Shores. Dr. Williams has asked me to forward this response to Tuesday's ALSWorldwide posting.
Blessings to each of you and thank you for your time.
Carl M. Brackett
March 19, 2013
March 12, 2013
We have discontinued treating ALS patients right now with the plan to move forward into a trial and more
advanced therapy in Colombia. I have initiated the gene therapy project. We are developing the gene therapy
into a Biotech Company. This will allow better opportunities for investors and hopefully allow us to move in the
direction of gaining FDA approval. The Biotech will be named Neuralgene, the website should be up within a
week. The gene therapy has several different modes of action, but the main targets are TDP-43 and Complement inhibition.
TDP-43 accumulation is toxic to neurons, this therapy helps eliminate it from the cells.
The gene therapy is designed to transfer the genes specifically to neural tissue (brain and spinal cord). The gene therapy will
have to be administered into the CSF via a catheter that is directed along the entire length of the spinal cord. This allows equal
gene delivery. It will take 4 weeks for gene expression to begin, and it will continue to increase until it hits a steady plateau at 4-6 months.
It is believed that the gene therapy may be life-long. Current studies have shown continuos gene expression now lasting up to 8 years+, from
a single treatment of AAV gene therapy.
In addition to the gene therapy treating the patients own cells, we will also use cultured stem cells as well. This should give us
benefits from the gene therapy and stem cells. We will be using cultured stem cells, which will increase the number of stem cells per treatment.
Though it is not fully confirmed, it looks as if we will be working with El Bosque University in Bogota, Colombia. I met with the team of doctors there who
are already doing stem cell research. Most of their work has been in Parkinson's. They have done some work with ALS. Their findings are much like the
findings that are seen in any stem cell treatment. It is beneficial for some patients, but results do not seem to be permanent. We feel the gene therapy
can make these results permanent.
At El Bosque, we plan to work in conjunction with Dr. Torres-Obando, a Neurologist, who has been leading their stem cell research. The University plans
to build a large research lab in the near future. The Precision StemCell clinic will be in a building at El Bosque (Clinica El Bosque). We plan to have the
new facility open in 2-3 months. Patients that are interested, we are putting on a waiting list.
We plan to move forward with the development of trials for the gene therapy, but we also plan to have a parallel path in Colombia, allowing access to
February 19, 2013
We had a 2 day visit from Stephen Byer, and ALS untangled. I don't know what the final opinion will be, but I feel certain
they must feel that we are trying to help ALS patients.I am on the agenda for the FDA February 25 meeting. The FDA has not approached me, however we are discontinuing treatments in the US except for those that will either be used with the cultured cell program in Bogota, or the future FDA trial.
I think they really need to get aggressive on incentives, which may catapult ALS drug and biological therapy development.
I arrived in Colombia last night. Everything is good here. We are continuing to move forward and are working on setting up an office here.
February 1, 2013
Precision Stemcell video. About 30 minutes long describing background and plans.
January 28, 2013
Sent in response to a note posted by Ted Harada:
Ted is correct that patients usually see results
within the first 3 months. We know that now, but did not originally. Initially, we thought as many did, that
regeneration of the nerves was key. We knew that nerve regeneration will take time. Now we understand that
it is substances that are produced by the stem cells which inhibit ALS. I feel that we have identified at least some of these substances.
Stem cells will produce them for a period of time, we don't know how long, then it seems (we believe) that this production
ceases or reduces to a non therapeutic level. Ed, as you know, we have a plan with a combination of gene therapy and
stem cells to make this production last longer (hopefully lifelong).
This therapy is advancing and we must work together.
Unfortunately, we are not a traded company, but we are applying for grants. We are also applying to become a sanctioned trial. Our funding is limited. As people are aware, we treat very
few patients, but we get calls and emails from plenty. The amount that the patients have been paying does not
cover my total related expenses.
I am just one person who stumbled into this. I was never planning on getting into ALS therapy.
I feel compelled to try to help this patients. Since I am a doctor, I do not know all the in and outs of
setting up a company, obtaining funding, etc. I certainly welcome any help.
I hope that we can all work together to move this therapy forward.
January 25, 2013
from Dr. Williams
See email.and These are the people we are trying to work with to get an official trial.
Right now they are waiting for me to send them a research protocol, so that it can
be reviewed by the IRB.
We will have to have certain patient criteria. Also the trial will have to be fully funded. Patients
will not be able to pay for any portion of the procedure if done on the trial.
We have been sending out info trying to find research grants. It is hard for me to handle
all this, plus my regular work. I think at some point soon, if I could find a PhD to work with me,
they could help with grant writing.
January 24, 2013
update from Angie, patient coordinator for Dr. Williams:
Good Morning Mr. Ed.
I do not have anyone on the schedule for March/April. I do believe that Mr. Paul is planning to come but We have not discussed dates. As for the cost it is per person.
TREATMENT OPTION 1
A single harvest one time stem cell injection done at our office in Gulf Shores, Alabama is $19,500.00 (USD)
TREATMENT OPTION 2(Columbia)
A double harvest, that includes treatment option 1 with a second treatment done in Bogata, Columbia. This option is $37,000.00 here in Alabama with additional charges to the patient in Bogata of $8,000.00 to $10,000.00 (USD)
Patients are responsible for all of their own travel expenses and accommodations.
January 19, 2013
I attended the Kevin Turner Foundation event in Montgomery, AL last night with the screening of
the documentary American Man. I also brought my wife and some of the office staff with me.
The documentary is powerful and emotional. I am sure there was not a dry eye in the house.
I would estimate there were about 1000 or more people who attended. Many prominent athletes and coaches were there as well.
I was glad that my wife attended, even though she is very understanding and supportive of my work,
this really hit home with her. I think now she has a better understanding
of what I have to do and why. People that have not been around ALS (and I only see it briefly myself), but
they do not know how devastating a disease it is for those (and their family) who have it. Believe me,
I think I now have a pass for consuming all my free moments working on this project.
The event was very successful. Though Kevin was busy, we spoke several times. Ed, I know he (Kevin) is
impossible to catch up with, but I stressed to him how important it is that we must all work together.
I think he will be getting with you soon. He is very excited about moving forward with Colombia, both
culturing and ultimately genetic engineered stem cells. He plans to be the first patient with the cultured
Ed, if you want to post this as an update, I think that should be fine.
January 16, 2013
update from Dr. Williams
I wanted to mention that the Kevin Turner Foundation event is this Friday evening (Jan 18) in Montgomery, Alabama. I certainly
hope that any patients in the area will try to attend.
Also, I will try to give some clarification related to the new treatments in Colombia.
The treatment in Colombia will involve the use of cultured autologous adipose derived stem cells. We plan to use the same technique for neural reprogramming.
The procedure will involve an initial harvest in Gulf Shores, Alabama. This will be a larger volume harvest than normal. This will allow us to process and
treat the patient on the same day (standard procedure) plus at a later date with the cultured stem cells (2nd procedure). Stem cells will be shipped to (Stem) in Bogota, Colombia. A larger harvest will have to be performed with some added processing to obtain enough stem cells for both culture and same day treatment. This will increase time and supplies.
The cultured (expanded) stem cells will take about 21 days at Stem in Bogota. There is the added expense of shipping and culture. Then after the culture (expansion) of cells is complete, both the patient and myself will travel to Colombia. I will perform the injections myself, the Colombian doctors will be with me for assistance. We will be using their facility, which is used for surgery/procedures. We are having to provide our own imaging.
The cultured stem cells produces on average 170+ million stem cells. These cells are produced under very strict guidelines, holding to all the highest standards of cell culture.
Stem has already engaged in several other human studies. The culturing of the cells holds several advantages over a same day procedure (performed only in the USA).
1) 170+ million stem cells versus an estimated 30 million. Studies indicate the more cells, the better the success. See recent Harvard study.
2) Stem cells can be stored and re-expanded (cultured) for addition injections without the need for harvesting. This means that a patient can have multiple injections with only going through the harvest procedure once.
3) Stem cells will be ready as the therapy advances. When new developments occur, it will be a shorter time frame to treatment, as the cells will be ready for culture.
Carl has been calculating the cost involved. He will send those figures to you. We have an idea on the harvest, shipping and culture. We do not know the cost of the injection in Colombia at this time, we are waiting on that information from the facility in Bogota. It is very challenging for us logistically to handle this procedure. Due to the expense involved,
I am not sure that we can treat more than 3 patients every 2-3 months. I know that it is expensive for the patient, but there are also numerous expenses that we will take on during this process. Due to this fact, I don't think I can afford to treat more at this time. We will also have to treat 3 patients at one time to reduce the overall related cost.
We were planning our first procedure (harvest and injection in Alabama) on Jan 21, which would mean the second procedure (in Colombia) would be the week of Feb 18. Unfortunately, a couple of patients had to cancel. If we can't fill the schedule, we will have to cancel this time. I will need to go to Colombia to work on the research project (in Feb), I won't be able to miss that much work close together, so it may be 2-3 months before we can try again.
In addition, we will probably need to cut back the number of patients that we treat in the USA (same day procedure). We currently average about 4 procedures a month. We will probably need to decrease that, maybe to 2, so that we can maintain our regular practice (other image guided procedures). Unfortunately, with lower reimbursement for our standard imaging procedures, we have to treat a higher volume of patients to keep up with overhead. This will cut into the number of stem cells cases, as they take a long time and displace a large number of regular cases. This will hopefully allow me to catch up on overhead. Once we get back in good shape, I will hopefully be able to help with funding on the stem cell/gene therapy project.
JasonJanuary 13, 2013
update from Dr. Williams
"We are planning to treat a limited number of patients, probably 3, with the new culturing technique. The patients will have an initial harvest performed at our clinic in Gulf Shores, AL. We will perform our standard treatment with the typical (approximate 30 million) stem cells obtained from a harvest. We will do an additional harvest which will be shipped to Stem in Bogota, Colombia. Stem will perform culturing of those cells, which should result in 170+ million stem cells. We plan to do this initial harvest on January 21. The administration of the cultured stem cells will be performed in Bogota, Colombia during the week of February 18. We are very excited about this first new step in significantly increasing stem cell numbers. Due to the extra expense related to this whole process, we are trying to group several patients together, that way the shipment can be performed at one time to keep cost down. We have purchased a special electronic temperature control shipping container, designed for stem cell shipping. Additional stem cells will be stored at the lab in Bogota. This will allow additional stem cell treatments in the future (theoretically it could be done every 3 weeks), without having to go through the harvest process again. We are still on standby for the gene therapy stem cell mouse study, pending funding. "
January 8, 2013
I am going to send you a copy of an article just released from researchers at Harvard. I find it re-assuring that their research supports the same ideas that I have.
They also agree that the stem cells are producing substances which inhibit ALS. They did conclude that they are not exactly sure which substances are helping. I will go a step further and say that….is the main one, but there are several others which are secondary. This supports the theory of our planned project….
I had a phone discussion with the people at prize4life. I discussed with them about the project. They are very excited and agree that this is a novel idea with great promise. One thing they told me, which was somewhat disappointing, was that even though they supply the mice for free, Northwestern University requires a $50k licensing fee. So, they really are not for free. In fact, to me, those are expense mice.
My Colombian counterparts are trying to get support from a local university (El Bosque). If they will work with us, we can use some of their lab and personnel, which will save some expense.….